Medications

Neuromyelitis optica spectrum disorder (NMO) is a rare inflammatory condition that may affect the eyes, spinal cord, and brain. Drug treatment for NMO has improved over recent years, and research continues to find more options for this condition.1

Drug treatment for NMO can be described as either acute (immediate or short-term) or preventative. Acute drugs are used to treat symptoms of acute attacks when they start and symptoms related to NMO attacks. Preventive drugs are used to prevent the damaging attacks of the disease from happening.2,3

Understanding these drugs, how are they used in NMO, and what additional options may be available can help you get the right treatment.

Acute drugs

These are prescribed to treat inflammation and symptoms related to attacks of NMO.3

Steroids

Steroids (corticosteroids) are strong anti-inflammatory drugs. Your body makes steroids normally. When given in doses larger than what your body typically makes, steroids quickly decrease inflammation.4

High-dose steroids are currently the only type of drug used in an acute attack to specifically treat the inflammation from NMO. These drugs help treat vision loss due to optic neuritis (inflammation of the nerve fibers in the eye). Methylprednisolone is the most common IV steroid given to those with NMO.1,3,5

NMO attacks may also cause inflammation of the spinal cord, known as transverse myelitis.High-dose steroids also help treat inflammation to the spinal cord that may occur with transverse myelitis.1,5

Preventative drugs

Prevention of NMO attacks is vital. Each attack can lead to increased damage to the brain, eyes, and spinal cord. The goal of preventative treatment is to decrease the number and severity of attacks. Until recently, preventive drugs were not available to those with NMO.6,7

In NMO, the antibody that attacks a protein called aquaporin-4 (AQP4) causes damage to the protective coating on nerve cells in the brain and spinal cord. This coating is known as myelin.8

The U.S. Food and Drug Administration (FDA) has approved 3 drugs for the treatment of those with AQP4 antibody (AQP4-IgG) NMO. These drugs are known as biologics and are made from living cells.2,7

Soliris (eculizumab)

Soliris is a laboratory-made antibody used to decrease the risk of an NMO attack. The exact way Soliris works is not known. Soliris is given by IB infusion, usually every 5 weeks.9,10

Uplizna™ (inebilizumab)

Uplizna™ is believed to work by lowering the number of your body’s B cells, which are part of your immune response. The goal of this drug is to decrease immune response and prevent further attacks on the central nervous system. Uplizna is given twice a year by IV infusion.11,12

Enspryng™ (satralizumab)

Enspryng™ is the first approved treatment in NMO that is made to block interleukin-6 (IL-6). IL-6 is a protein made by cells in the body and plays a role in the inflammation that occurs in NMO. Enspryng blocks IL-6 from attaching to the cell surface. This stops the inflammation cascade and prevents autoimmune attacks.13,14

Enspryng is given by self-injection once per month. If your doctor prescribes this drug, you will receive injection training on the safe and correct way to inject it.13,14

Other preventative drugs

Off-label uses may be common in rare diseases where not many drugs are used to treat a condition. If you and your doctor decide on off-label use, it is important to understand that the FDA has not yet determined the drug is safe when used the way you are taking it. However, this does not mean your use of the drug is unsafe. Often, it just means there has not been a large, official clinical trial of this specific drug for this specific rare disease. Talk to your doctor about your treatment questions and needs.15

Some off-label and unapproved drugs commonly used to treat NMO include:16-22

  • Rituxan (rituximab), a laboratory-made antibody
  • Mitoxantrone, an anti-cancer drug
  • Campath and Lemtrada (alemtuzumab), which are laboratory-made antibodies
  • Imuran and Azasan (azathioprine), which are drugs that decrease the immune response
  • CellCept (mycophenolate mofetil), a drug that decreases the immune response
  • Trexall, Otrexup, and Rasuvo (methotrexate), which are disease-modifying antirheumatic drugs (DMARDs)
  • Actemra (tocilizumab), a laboratory-made antibody

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Written by: Katie Murphy | Last reviewed: September 2021